Expression of Aromatic Amino Acid Decarboxylase and Vesicular Monomine Transporter Using Lentiviral Vectors to Prevent L-Dopa Induced Dyskinesias

Leff, Stuart, Ph.D.
Emory University, Atlanta, GA

Most PD patients develop fluctuations and /or abnormal involuntary movements(AIMs) over time despite the symptomatic use of the “gold standard,” Sinemet.  Such complications only seem to develop on the intermittent dosing used by human patients, but not in laboratory animals that have been treated via continuous drug delivery methods.  Dr. Stuart Leff and his colleagues at Emory University will use parkinsonian rats in their study of
the co-delivery via injected lentiviral vectors of aromatic amino acid decarboxylase (AADC) and vesicular monoamine transporter (VMAT-2).  Their hypothesis is that these two enzymes are needed to buffer and prolong the release of administered dopamine over time which would preclude the dyskinesias so commonly experienced by patients. This study is an excellent example of the outgrowths of gene therapy work in many laboratories.